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2016: Progress in Putting Patients First

As the lights go out on the year and we raise our glasses to cheer the arrival of 2017, Rx4good looks back at the top strides made in 2016 in putting patients first.

Walking the Talk, Oncology Leads the Way

If major meetings are a bellwether of a commitment that’s here to stay, ASCO and ESMO were evidence that patients are moving from the sidelines to the center. ASCO’s 2016 theme was Collective Wisdom: The Future of Patient-Centered Care and Research, and program topics spanned important patient issues from financial toxicity to shared decision-making. ASCO has included patient advocates as members for a number of years offering them specific membership benefits. Going a step further, ESMO announced in October that it was recognizing patient advocates as healthcare professionals, opening its membership to them and giving them full voting rights and access.

Patients Included, an organization that has been pushing for patient involvement in major conferences by offering certification and a “Patients Included” seal of approval since 2010, stepped up its efforts and certified 22 organizations during 2016, including PCORI. Let’s hope this trend continues into 2017 with more organizations truly integrating the patient voice into their meetings.

FDA Adding Rigor to Patient Engagement

Since 2013, the FDA has held 20 of 24 disease-specific Patient Focused Drug Development meetings. Now FDA is looking further at how to institutionalize the patient perspective. At an EyeforPharma Patient Centered Clinical Trials conference in November, Dr. John Whyte, Director of Professional Affairs and Stakeholder Engagement at the FDA said, “The whole point of patient engagement is utilizing patients’ unique expertise to determine what is clinically meaningful to them and how they evaluate risk vs benefit. It’s not about finding 100 or 1,000 patients and simply talking at them. It’s about doing research around patient engagement in a methodologically rigorous way, which is reproducible and also representative of patients.” Here are FDA plans for integrating the patient:

Between 2017 and 2021, the agency expects to hire additional staff focused on engaging with patients and facilitating the development and use of patient-focused drug development methods.

In 2019, the agency plans to issue draft guidance describing how companies can collect information from patients, and how that information can be used in the drug development and regulatory decision-making process.

Another draft guidance describing how meaningful patient perspectives and information can be collected in clinical trials is expected in 2020.

And finally, a draft guidance on patient-reported outcome measures to replace the one released in 2009 is set to be available come 2021.

It promises to be an interesting five years!

Data Sharing Speeding Safe Therapies to Patients

This year the big story was more sharing of big data.

In June, Vice President Joe Biden, who leads President Barack Obama’s Cancer Moonshot initiative, announced that the National Cancer Institute would launch an open-access database containing anonymous genomic and clinical data from 12,000 patients that any scientist or doctor can tap into for insights on the molecular makeup of many cancer types.

Transcelerate announced a Placebo and Standard of Care (PSoC) Initiative, with the potential to significantly reduce the number of patients enrolled in clinical trials by allowing the reuse of data from previous studies. In decreasing the time spent on cumulative trial execution, the hope for PSoC is speeding therapies to patients in need.

OpenTrials, a “centralised home” for all clinical trials information, announced that it will extract and match data from a number of sources and registries including PubMed, ClinicalTrials.gov and Drugs@FDA, as well as from the EU Clinical Trials Register, the UK’s Health Research Authority, and the World Health Organization’s International Clinical Trials Registry Platform, to make trial data more accessible and transparent.

IBM Watson Health and Celgene announced a collaboration to develop a new platform for evaluating the safety of drugs—both before and after they hit the market. “Watson for Patient Safety,” will sift through medical records, claims data, and millions of electronic submissions to the FDA about potential drug side effects to see if it can learn about the hidden dangers of medicines earlier in a drug’s lifecycle.

Companies Organize Around the Patient

Companies stepped up their efforts to organize around patients this year, with more naming chief patient advocates or heads of patient-centricity in posts around the world to help embed patient-centric thinking across the organization. At last count, at least seven biopharma companies have named someone to this role, among them: AstraZeneca, Pfizer, Sanofi, Merck, Amgen, Amicus, UCB.

Finally, this year EyeforPharma, under the umbrella of the Aurora Project, published a white paper “Patient Centric Profitability” based on global research with 2,300 respondents from 84 countries. The researchers asked whether companies could be patient-centric and profitable at the same time. We hope that more conversation occurs around this topic in 2017 as companies realize the business value of including patients in their decisions.

While there is much work that remains to be done, we have made progress–both as an industry and as a society–in elevating the patient voice and perspective to change the systems that are meant to serve them. We at Rx4good believe that this trend will continue, and our 2017 resolution is to continue to do our part.